Rare diseases

R&D

Net Treatment Benefit in rare disease trials: Aligning trial...

Net Treatment Benefit provides a formal framework to integrate multiple prioritised outcomes into a single measure of overall treatment effect.

News

NHS "isn't delivering equitable care for rare diseases"

A Genetic Alliance UK report claims the NHS is failing to meet the needs of many of the 3.5 million people in the country who live with rare diseases.

R&D

How innovative trial approaches are advancing rare disease r...

Innovative trial designs that harness data augmentation, RWD, and the power of AI are enabling a new era of rare disease research.

News

US lawmakers take FDA to task over rare diseases record

Lawmakers and patient advocates in the US are concerned that the FDA's regulation of rare disease therapies has been inconsistent and overly cautious.

Market Access

What rare disease commercialisation in Europe teaches us - a...

Rare disease launches are the ultimate stress test for European commercialisation.

Oncology

Collaboration & personalisation in rare disease R&D, with AO...

For Rare Disease Day, in an AOP Health-sponsored podcast Melissa Fellner, VP of Global Therapeutic Areas, Commercial Operations, discussed their work.

Rare diseases

Editor's Picks